STX209 (arbaclofen) for autism spectrum disorders: an 8-week open-label study.
STX209 looked helpful for irritability in an open trial, but firmer evidence is needed before choosing it over proven options.
01Research in Context
What this study did
Doctors gave kids with autism a pill called STX209 every day for eight weeks. Parents and clinicians filled out rating scales about irritability and social withdrawal before and after. No placebo group was used.
What they found
Both parents and clinicians reported less irritability and fewer social withdrawal behaviors at week eight. The drug was generally well tolerated with no major safety red flags.
How this fits with other research
Capio et al. (2013) ran a similar-length study but added a placebo arm and found large irritability drops only with higher-dose risperidone. Their controlled design shows the open-label gains seen here might shrink under placebo testing.
Rezaei et al. (2018) combined risperidone with PRT and saw medium aberrant-behavior drops, hinting that pairing meds with ABA could beat meds alone. That team’s work suggests future STX209 trials may want to add behavioral treatment.
Hadjikhani et al. (2015) also used an open-label pill design and saw social gains with bumetanide. Both pilots flag the same next step: run randomized, placebo-controlled studies before clinical use.
Why it matters
The pill eased irritability quickly, something families often request. Yet without a placebo group you cannot tell if the gains came from the drug or from extra attention. Share these hopeful but early data with prescribers, keep measuring behavior on your own scales, and stay ready to fold STX209 into a combined plan if later RCTs confirm benefit.
Want CEUs on This Topic?
The ABA Clubhouse has 60+ free CEUs — live every Wednesday. Ethics, supervision & clinical topics.
Join Free →Track irritability on a simple 0–10 scale before and after any med change and share the data with the prescribing doctor.
02At a glance
03Original abstract
STX209 (arbaclofen), a selective GABA-B agonist, is hypothesized to modulate the balance of excitatory to inhibitory neurotransmission, and has shown preliminary evidence of benefit in fragile X syndrome. We evaluated its safety, tolerability, and efficacy in non-syndromic autism spectrum disorders, in an 8-week open-label trial enrolling 32 children and adolescents with either Autistic Disorder or Pervasive Developmental Disorder-Not Otherwise Specified, and a score ≥17 on the Aberrant Behavior Checklist (ABC)-Irritability subscale. STX209 was generally well-tolerated. The most common adverse events were agitation and irritability, which typically resolved without dose changes, and were often felt to represent spontaneous variation in underlying symptoms. Improvements were observed on several outcome measures in this exploratory trial, including the ABC-Irritability (the primary endpoint) and the Lethargy/Social Withdrawal subscales, the Social Responsiveness Scale, the CY-BOCS-PDD, and clinical global impression scales. Placebo-controlled study of STX209 is warranted.
Journal of autism and developmental disorders, 2014 · doi:10.1007/s10803-013-1963-z